PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS] (PRECISE)

A Comparative Observational Study to Evaluate the Safety and Effectiveness of Xromi (Hydroxycarbamide Oral Solution 100mg/ml) for the Prevention of Vaso-occlusive Complications of Sickle Cell Disease in Children Under 2 Years of Age.

This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).

The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Drug: Xromi

Detailed Description

The PRECISE study is a combined Post-Authorisation Safety Study (PASS) (Category 3) and a Post-Authorisation Efficacy Study that aims to provide data on the safety and effectiveness of hydroxycarbamide 100mg/ml oral solution (Xromi ®) administered prospectively to children under 2 years of age, over a follow-up period of 24 months compared to matched retrospective comparators who were treatment naïve.

This is a non-interventional, matched cohort study involving children with SCD aged 9 to under 24 months. The study comprises two groups:

• A prospective Xromi®-exposed cohort, enrolled at the time of treatment initiation and followed for 24 months.
• A retrospective comparator cohort, matched 2:1 by site, age, and β-globin genotype, identified from clinical records of children not treated with hydroxycarbamide at the index date.

The primary objective is to compare the incidence of adverse events of special interest (AESIs) between the two cohorts. Secondary analyses will assess the comparative effectiveness of Xromi® on clinical events, laboratory parameters, and physiological assessments. Exploratory analyses will examine treatment-related safety and effectiveness by dose, subgroups, and exposure to hydroxycarbamide during follow-up.

Data will be sourced from routine clinical practice through chart reviews and follow-up visits. No study-specific interventions will be introduced. The study is planned across specialist sites in the UK and Germany, with potential expansion to other European countries if recruitment targets require.

• Study Type: Observational
• Enrollment (Estimated): 180

Contacts and Locations

• Study Contact: Name: Hussain Mulla, PhD; Phone Number: +44 (0)116 223 0100; Email: hussain.mulla@novalabs.co.uk
• Study Contact Backup: Name: Sarah Edwards, PhD; Phone Number: +44 (0)116 223 0100; Email: sarah.edwards@novalabs.co.uk

Study Locations

• Germany
  • Hamburg, Germany
    • Recruiting
    • Universitätsklinikum Hamburg-Eppendorf
  • Heidelberg, Germany
    • Recruiting
    • Universitätsklinikum Heidelberg
• United Kingdom
  • Basildon, United Kingdom
    • Recruiting
    • Basildon University Hospital
  • Cardiff, United Kingdom
    • Recruiting
    • Noah's Ark Children's Hospital for Wales
  • London, United Kingdom
    • Recruiting
    • Kings College Hospital
  • London, United Kingdom
    • Recruiting
    • University College London Hospital
  • London, United Kingdom
    • Recruiting
    • Evelina London Children's Hospital
  • London, United Kingdom
    • Recruiting
    • The Royal London Hospital
  • London, United Kingdom
    • Recruiting
    • North Middlesex University Hospital
  • London, United Kingdom
    • Recruiting
    • Whittington Hospital
  • Manchester, United Kingdom
    • Recruiting
    • Royal Manchester Children's Hospital
  • Oxford, United Kingdom
    • Recruiting
    • John Radcliffe Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of children with SCD who meet all the inclusion criteria and none of the exclusion criteria.

Identification of participants will be conducted as follows:

• Prospective exposure cohort: Potential participants will be identified as they attend the participating sites where eligibility criteria will be assessed prior to initiation with Xromi.
• Retrospective comparator cohort: Potential participants will be selected from clinical chart review for hydroxycarbamide naive children that could be matched to prospective participants.

Description

Prospective Exposure Cohort

• Inclusion criteria:

  • Aged from 9 months to under 2 years at the index date.
  • Diagnosis of SCD.
  • Known β-globin genotype at the index date.
  • Prescribed Xromi® for the prevention of complications of SCD.
  • Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies.

• Exclusion criteria:

  • Previous use of hydroxycarbamide of any formulation before the index date.
  • Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date.
  • Known hypersensitivity to any of the excipients of Xromi® at the index date.
  • Contraindications to the drug at the index date: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (creatinine clearance: CrCl <30 ml/min); presence of at least one of the following: Absolute neutrophil count (ANC) < 1.0 x 10^9/L, absolute reticulocyte count (ARC) <80 x 10^9/L, platelets <80 x 10^9/L.
  • Participating in another clinical study of an investigational medicinal product (IMP) at the index date.
  • Anti-retroviral medicinal products for human immunodeficiency virus (HIV) at the index date.
  • Active malignancy at the index date.

Participants in the prospective exposure cohort who are prescribed Xromi® but do not initiate treatment will be excluded from the dataset.

Retrospective Comparator cohort

• Inclusion criteria:

  • Aged from 9 months to under 2 years at the index date.
  • Diagnosis of SCD.
  • Known β-globin genotype.
  • Matched to an exposed participant.
  • Parent(s) (or a legal representative(s)) provides written informed consent to participate in the study, unless there is a waiver, non-opposition, or blanket written informed consent by the parent for research studies.

• Exclusion criteria:

  • Use of hydroxycarbamide of any formulation before or at the index date.
  • Receiving regular blood transfusions (occurring every 8 weeks or more frequently) at the index date.
  • Presence at the index date of any of the following: severe hepatic impairment (Child-Pugh classification C); severe renal impairment (CrCl <30 ml/min); presence of at least one of the following: ANC < 1.0 x 10^9/L, ARC < 80 x 10^9/L, platelets < 80 x 10^9/L).
  • Participating in another clinical study of an IMP at the index date.
  • Anti-retroviral medicinal products for HIV at the index date.
  • Active malignancy at the index date.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Prospective Exposure Cohort; Children with SCD aged 9 months to under 2 years of age who are newly prescribed Xromi®, will be identified prospectively. These participants will be followed up for 24 months from their index date, regardless of whether they continue treatment with Xromi®, discontinue all hydroxycarbamide treatment, or switch to another formulation of hydroxycarbamide. The decision to prescribe Xromi® will be made solely by the physician independently of the study, as part of standard care. -	Drug: Xromi; Xromi is indicated for the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 9 months of age as part of standard clinical practice; Other Names: hydroxycarbamide oral solution 100mg/ml; liquid hydroxyurea; LO1XX05
Retrospective Comparator Cohort; Children with SCD and naïve to any hydroxycarbamide formulation at the index date. These participants will be identified retrospectively using the data from the last 10 years up to the date Xromi® was first used in children from 9 months to under 2 years of age at each individual site. The 24-month follow-up will be retrospective from the date they are matched to the exposed participant, irrespective of whether they start on any formulation of hydroxycarbamide during the follow-up.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
AESI - Myelosuppression (Neutropenia)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of neutropenia is ANC <1.0 x 10^9/L	Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Reticulocytopenia)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of reticulocytopenia is ARC <80 x 10^9/L, unless Hb >90 g/L,	Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Thrombocytopenia)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of thrombocytopenia defined as platelets <80 x 10^9/L.	Pre-baseline, Baseline to 24 months
AESI - Myelosuppression (Anaemia)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Myelosuppression protocol definition of anaemia defined as Hb <45 g/L.	Pre-baseline, Baseline to 24 months
AESI - Abnormal Weight Gain	Weight (kg) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines	Baseline to 24 months
AESI - Abnormal Weight Loss	Weight (kg) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines	Baseline to 24 months
AESI - Increase in Hepatic Enzyme (ALT)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and normal laboratory ranges. Defined as an increase in hepatic enzyme Alanine transaminase (ALT) increased (U/L)	Baseline to 24 months
AESI - Increase in Hepatic Enzyme (AST)	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and normal laboratory ranges. Defined as an increase in hepatic enzyme Aspartate transaminase (AST) increased (U/L)	Baseline to 24 months
AESI - Alopecia	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of alopecia (a skin and subcutaneous tissue disorder).	Pre-baseline, Baseline to 24 months
AESI - Other Hair Loss	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of other hair loss (a skin and subcutaneous tissue disorder).	Pre-baseline, Baseline to 24 months
AESI - Skin Hyperpigmentation	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of skin hyperpigmentation, including oral and nail hyperpigmentation.(a skin and subcutaneous tissue disorder).	Pre-baseline, Baseline to 24 months
AESI - Rash	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of a rash (a skin and subcutaneous tissue disorder).	Pre-baseline, Baseline to 24 months
AESI - Skin Ulcers	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in study protocol as presence of skin ulcer, including leg ulcer (a skin and subcutaneous tissue disorder).	Pre-baseline, Baseline to 24 months
AESI - Growth Retardation	height/length (cm) Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort and standard care guidelines (as a drop of 2 or more centiles on growth charts over time).	Pre-baseline, Baseline to 24 months
AESI - Bacterial Infection	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated bacterial infection.	Pre-baseline, Baseline to 24 months
AESI - Viral Infection	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated viral infection.	Pre-baseline, Baseline to 24 months
AESI - Fungal Infection	Incidence of adverse events of special interest (AESIs) in children who have SCD and are treated with Xromi®, in comparison to a retrospective comparator cohort. Defined in the protocol as a proven or treated fungal infection.	Pre-baseline, Baseline to 24 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Other Adverse Events	Occurrence of other adverse events (AE), adverse reactions (AR) and serious adverse events (SAE) or serious adverse reactions (SAR) will be collected during follow-up.	Baseline to 24 months
Painful Vaso-occlusive Crisis (VOC)	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. - VOC events will include dactylitis.	Pre-baseline, Baseline to 24 months
Acute Chest Syndrome (ACS)	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Splenomegaly	Occurrence of the clinical event Splenomegaly will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Priapism	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Baseline to 24 months
Hepatobiliary disorder	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Splenic Sequestration Crisis	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Surgery	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. Defined in the protocol as any surgery that is planned, emergency, or due to pre-existing conditions e.g. SCD.	Pre-baseline, Baseline to 24 months
Blood Transfusion	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Cerebrovascular accident	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. The protocol definition includes silent stroke	Pre-baseline, Baseline to 24 months
Abnormal or Conditional TCD	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. - Transcranial Doppler Scan (TCD)	Pre-baseline, Baseline to 24 months
Hospitalisations for SCD	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. The reason for hospitalisation and duration will be collected.	Pre-baseline, Baseline to 24 months
Non-hospitalised Visit for SCD	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®. Protocol definition includes Emergency department (ED) visits/treatment centres/paediatric ward/day unit attendances for SCD	Pre-baseline, Baseline to 24 months
Other Clinical Events	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator to compare the Effectiveness of Xromi®.	Pre-baseline, Baseline to 24 months
Other Event - Death	Occurrence of the clinical event will be collected during follow-up in both cohorts by the investigator. Protocol definition includes any death occurring in the study both related and un-related to Xromi treatment	Baseline to 24 months
Haemoglobin (Hb)	(g/L) or (g/dl) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Foetal Haemoglobin (HbF)	(%) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Haemoglobin Fractions	(%) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected. - Haemoglobinopathy screen results including: HbS, HbA, HbA2, HbC	Pre-baseline, Baseline to 24 months
Absolute Neutrophil Count (ANC)	(10^9/L or /nL) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Absolute Reticulocyte Count (ARC)	(10^9/L or /nL) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Platelet Count	(10^9/L or /nL) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
White Blood Cell Count (WBC)	(10^9/L or /nL) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Mean Corpuscular Volume (MCV)	(fl) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Mean Corpuscular Haemoglobin (MCH)	(pg) Haematological Parameter. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Ferritin	(µg/L) Iron Profile. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Transferrin Saturation	(%) Iron Profile. Iron binding saturation or transferrin saturation. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Alanine Transaminase (ALT)	(U/L) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Alkaline Phosphatase (ALP)	(U/L) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Aspartate Transaminase (AST)	(U/L) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Total Bilirubin	(µmol/L or mg/dl) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Lactate Dehydrogenase	(U/L) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Gamma-Glutamyl Transferase (GGT)	(U/L) Liver Function tests. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Serum Creatinine	(µmol/L or mg/dl) Renal function test. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Estimated Glomular Filtration Rate (eGFR)	(ml/min/1.73m^2) Renal function test. Calculated value, Interpreted in line with UK CKD guidelines from creatinine values. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Albumin-Creatinine Ratio (ACR)	(Calculated value). Renal function test. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Liver Size	(cm) from costal margin. Physiological Assessment. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Spleen Size	(cm) from costal margin. Physiological Assessment. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months
Maximum Time Averaged Mean Velocity (TAMV)	(cm/s) Cardiovascular function, assessed using transcranial doppler scan velocities. Results and dates of laboratory tests and physiological assessments will be collected, whenever available. At baseline, the most recent results, prior to Xromi® initiation in the prospective exposure cohort, will be collected. Results from laboratory tests and physiological assessments conducted during follow-up will also be collected.	Pre-baseline, Baseline to 24 months

Collaborators and Investigators

• Sponsor: Nova Laboratories Limited
• Collaborators: OXON Epidemiology
• Investigators: Study Director: Hussain Dr Mulla, PhD, Nova Laboratories Ltd.; Principal Investigator: Sara Dr Trompeter, MD, University College London Hospitals

Publications and helpful links

General Publications

• Thompson BW, Miller ST, Rogers ZR, Rees RC, Ware RE, Waclawiw MA, Iyer RV, Casella JF, Luchtman-Jones L, Rana S, Thornburg CD, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik S, Howard TH, Luck L, Wang WC. The pediatric hydroxyurea phase III clinical trial (BABY HUG): challenges of study design. Pediatr Blood Cancer. 2010 Feb;54(2):250-5. doi: 10.1002/pbc.22269.
• Rankine-Mullings A, Keenan R, Chakravorty S, Inusa B, Telfer P, Velangi M, Ware RE, Moss JJ, Lloyd AL, Edwards S, Mulla H. Efficacy, safety, and pharmacokinetics of a new, ready-to-use, liquid hydroxyurea in children with sickle cell anemia. Blood Adv. 2023 Aug 22;7(16):4319-4322. doi: 10.1182/bloodadvances.2023010099. No abstract available.

Study record dates

Study Major Dates

• Study Start (Actual): June 9, 2025
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): June 1, 2029

Study Registration Dates

• First Submitted: April 3, 2025
• First Submitted That Met QC Criteria: April 3, 2025
• First Posted (Actual): April 11, 2025

Study Record Updates

• Last Update Posted (Actual): March 5, 2026
• Last Update Submitted That Met QC Criteria: March 3, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: children; Hydroxyurea; Sickle Cell Disease; Sickle Cell Anemia; paediatric; Hemoglobin S Disease; Hydroxycarbamide; HbS Disease; Sickle Cell Disorders; Sickling Disorder Due to Hemoglobin S; hemoglobinopathy; Xromi; liquid formulation; hemaglobinopathy in children; paediatric formuation
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; Hemoglobinopathies; Organic Chemicals; Amides; Urea; Hydroxyurea
• Other Study ID Numbers: NOVDD-001; EUPAS1000000076 (Other Identifier: HMA-EMA RWD Catalogues); CHIL 62837 (Other Identifier: NIHR CPMS); 334976 (Other Identifier: HRA IRAS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

The study has been registered on a public database HMA-EMA RWD Catalogues and the study protocol has been shared publicly (EUPAS1000000076).

Study publication of aggregated outcome results on Clinical trials.gov and in Multi-centre study publication in relevant medical journal.

Investigators:

All Investigators will be provided with their individual participant data at the end of the study as eCRFs.

Regulatory Authorities:

The study protocol was shared with the EMA and MHRA. The draft SAP was shared with the EMA during initial review of the study protocol prior to study initiation.

CSR will be made available to the EMA and MHRA at the end of the study.

• IPD Sharing Time Frame: March 2025 - upto 25 years from end of study.
• IPD Sharing Access Criteria: Publicly accessible study protocol via the HMA-EMA RWD Catalogues and the study protocol has been shared publicly (EUPAS1000000076) Publicly accessible aggregated study results via ClinicalTrials.Gov.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No